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About the Regulatory Profession

The regulatory function is vital in making safe and effective healthcare products available worldwide. Individuals who ensure regulatory compliance and prepare submissions, as well as those whose main job function is clinical affairs or quality assurance are all considered regulatory professionals.

Regulatory Code of Ethics

One of our most valuable contributions to the profession is the Regulatory Code of Ethics. The Code of Ethics provides regulatory professionals with core values that hold them to the highest standards of professional conduct.

Regulatory Competency Framework

Like all professions, regulatory is based on a shared set of competencies. The Regulatory Competency Framework describes the essential elements of what is required of regulatory professionals at four major career and professional levels.

Regulatory Convergence

Join the brightest minds in regulatory at the annual Regulatory Convergence. See the global regulatory community in action. Intensive workshops. Topical sessions. Meet ups with regulators. This is where it all comes together.

Are Medical Countermeasures Meeting the Potential Needs of Children?

Is the US government doing enough to protect children from the threat of a medical emergency caused by a chemical, biological, radiological or nuclear accident? Not as much as it could be, concludes a new report just published by the Government Accountability Office (GAO), an intra-government watchdog that regularly assesses programs for deficiencies.

Background

Since the events of September 2011, federal agencies, and particularly the US Food and Drug Administration (FDA), have been working to strengthen the US' capacity to respond to a catastrophic event.

Take the following hypothetical: A small nuclear "dirty" bomb goes off in a city, severely injuring 10,000 people, the majority of whom have burn-related injuries, requiring hospitalization and immediate treatment. How do you treat these patients?

Under normal circumstances, that's a no-brainer: You take them to the burn unit of the local hospital. But that's not an option when there are 10,000 patients. No hospital can accommodate that many burn patients. In fact, the entire burn unit capacity of the US is just 1,850 beds, roughly half of which are occupied at any one time, and most of which are geographically out of reach for burn patients who might need immediate treatment.

So how can regulators respond to such a-thankfully hypothetical-scenario? Through careful, advanced planning involving a huge number of stakeholders.

A recent January 2013 meeting of health officials involved in the Public Health Emergency Medical Countermeasures Enterprise (PHEMCE), for example, worked together to try to get a new regulatory paradigm in order to allow for the development of medical devices and other therapies that could be used by most anyone to help treat burn victims.

Regulators have floated the idea of forming a pathway similar in philosophy to the Animal Efficacy Rule ("Animal Rule") pathway that now permits products to be approved based largely on just safety testing in humans while relying on other models, such as animal-based ones, for efficacy data.

Ethical Issues

But the same scenario has another question associated with it: What if those patients are children? Will they be given the same standard of care as adults?

To GAO, data show that the government hasn't been pushing hard enough to get pediatric medical countermeasures developed.

Explains GAO: "According to the Department of Health and Human Services (HHS), about 60 percent of the chemical, biological, radiological, and nuclear (CBRN) medical countermeasures in the Strategic National Stockpile (SNS) have been approved for children, but in many instances approval is limited to specific age groups. In addition, about 40 percent of the CBRN countermeasures have not been approved for any pediatric use. Furthermore, some of the countermeasures have not been approved to treat individuals for the specific indications for which they have been stockpiled."

Key Differences in Children Present Challenges

The main problem lies in the physiological differences inherent in children, who are-despite their appearances-not simply shorter, less emotionally mature versions of adults. Rather, pediatric populations have real differences that can cause medications to be metabolized differently or generate unique adverse events. While these problems have largely (if incompletely) been addressed in normal medical therapies for which there is an active need, medical countermeasures, which do not have an active need, present a unique ethical dilemma.

That is, how do you make sure a product is safe for use in a child if that same child is unlikely to ever benefit from the product? The Presidential Commission for the Study of Bioethical Issues is currently studying the same issue, noting that it is "particularly interested in policies, practices, research, and perspectives on ethical issues associated with pre- and post-event studies testing the safety, dose, and/or immunogenicity of medical countermeasures for and with children."

Specifically, the commission said it is interested in determining a conceptual framework through which it can consider "risk and societal value when reviewing pediatric clinical research in general and for medical countermeasures in particular." It wants that framework to include different types of information, criteria for proposed studies, ethical issues, scientific and public health strategies, communication strategies and the role of communities in designing and supporting research.

Real-World Challenges

And the lack of an ethical framework has real-world challenges to potential pediatric victims of a disaster. Take for example an adsorbed anthrax vaccine (ADA), a product developed in the aftermath of terror attacks in 2001 that sent the anthrax virus through the US postal system.

This particular vaccine lacks "sufficient data" to supports its use, GAO said, meaning that it would need to be administered to children under an investigational new drug protocol. While those protocols exist, it would require parents to essentially enroll their child into a modified clinical trial, further requiring a parent or guardian to sign a consent form before their child could receive the vaccine.

In the wake of a disaster, might a child's parents be unaccounted for and unable to consent? &nbsp;It's a troubling question, to be sure.

Interim Steps

But in the meantime, GAO's report makes the case that the government could be doing more to support the needs of children. Just seven out of the 12 emergency response plans reviewed by GAO included information about pediatric medical countermeasures and how to dispense them.

FDA and the Centers for Disease Control and Prevention (CDC) both received passive praise from GAO's report, which noted that they had taken the lead in dispensing information to state authorities on how to receive, distribute and dispense pediatric medical countermeasures. That strategy seemed to be one that GAO favored.

GAO also took note of the myriad of regulatory hurdles facing FDA and industry in the approval of medical countermeasures, explaining that FDA had identified the complex differences present in children relative to adults and the difficulty of extrapolating data between different age groups and animal species. "This is not straightforward," GAO bluntly added.

DHHS meanwhile said that it agreed with the report's characterization of the current state of pediatric development, and "emphasized that the needs of the pediatric population have been a priority for HHS and that the department is continuously progressing in this area."